NCT00003966

Defibrotide in Treating Patients With Liver Damage Following Peripheral Stem Cell Transplantation

Defibrotide for Hematopoietic Stem Cell Transplant Patients With Severe Hepatic Venocclusive Disease: A Phase I/II Study to Determine the Minimal Effective Dose

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 151 (actual)
• Sponsor: Dana-Farber Cancer Institute · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

RATIONALE: Giving defibrotide may be an effective treatment for liver damage that may result following peripheral stem cell transplantation. PURPOSE: This randomized phase II trial is studying defibrotide to see how well it works in treating patients with severe liver disease after undergoing peripheral stem cell transplantation.

Detailed description

OBJECTIVES: * Determine complete response rate in post-hematopoietic stem cell transplant patients with severe veno-occlusive disease of the liver treated with defibrotide. * Determine the minimal effective dose of this drug in these patients. * Assess toxicity and adverse side effects of this drug in these patients. OUTLINE: This is a randomized, multicenter study. All patients initially receive the same dose of defibrotide IV over 2 hours every 6 hours on day 1. On day 2, patients are randomized to 1 of 2 doses of defibrotide. * Arm I: On days 2-14, patients receive a lower dose of defibrotide IV over 2 hours every 6 hours. * Arm II: On days 2-14, patients receive a higher dose of defibrotide IV over 2 hours every 6 hours. In both arms, courses repeat every 14 days in the absence of disease progression or unacceptable toxicity. PROJECTED ACCRUAL: A total of 140 patients (70 per treatment arm) will be accrued for this study.

Conditions

• Veno-occlusive Disease

Interventions

Type	Name	Description
DRUG	defibrotide

Timeline

• Start date: 2000-04-01
• Primary completion: 2006-02-01
• Completion: 2006-04-01
• First posted: 2003-01-27
• Last updated: 2017-01-20

Locations

9 sites across 1 country: United States